Pegunigalsidase now challenges Fabrazyme's two-decade lead, while oral migalastat works for only 35 to 50% of Fabry patients.

Fabry disease is an X-linked lysosomal storage disorder caused by GLA mutations, leading to progressive renal, cardiac and cerebrovascular disease. Three FDA-approved agents span two mechanisms: two intravenous enzyme replacement therapies, agalsidase beta (Fabrazyme, approved 2003) and pegunigalsidase alfa (Elfabrio, approved May 2023), and the oral chaperone migalastat (Galafold, approved 2018), indicated only for patients with an amenable GLA mutation.

Migalastat offers oral, every-other-day dosing instead of biweekly infusion, but only for the roughly 35 to 50% of patients whose mutation is amenable, confirmed by a validated cell-based assay. The ATTRACT trial showed migalastat gave renal outcomes comparable to ERT in experienced patients. Pegunigalsidase's BALANCE trial demonstrated non-inferiority to agalsidase beta on eGFR slope over two years, giving prescribers a second IV option against an incumbent with no US biosimilar and a WAC near $250,000 to $350,000.

A genetic assay, not physician preference, decides who can even take the oral option.

Five questions this report answers:

Q1 - How do the two IV enzyme replacement therapies and the oral chaperone differentiate on mechanism and eligibility?

Q2 - What does pegunigalsidase's BALANCE non-inferiority result mean for switching from the Fabrazyme incumbent?

Q3 - Which patients qualify for oral migalastat, and how does the amenable-mutation assay define that population?

Q4 - Why has agalsidase beta held its roughly $250,000 to $350,000 price with no US biosimilar?

Q5 - What did the ATTRACT and FACETS trials show for migalastat in switch versus treatment-naive patients?

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